For Patients & Caregivers

HEMLIBRA Clinical Trial Results

Pivotal Trial Data
Long-Term Data
Other HEMLIBRA Clinical Trials

Proven Efficacy Across a Broad Range of Patients

Pivotal clinical trial program—the largest in hemophilia A for patients with and without FVIII inhibitors1,3

HEMLIBRA CLINICAL TRIAL DATA (PRIMARY ANALYSIS AND PRIMARY ENDPOINT OF TRIALS)

HAVEN 1: ADULTS AND ADOLESCENTS WITH
FVIII INHIBITORS

HEMLIBRA® (emicizumab-kxwh) HAVEN 1-4 Long-term Data

of patients taking HEMLIBRA ONCE A WEEK had 0 treated bleeds (n=35) (95% CI: 44.9; 78.5)1

HEMLIBRA® (emicizumab-kxwh) HAVEN 1-4 Long-term Data

of patients taking bypassing agents to treat bleeds on-demand (no prophylaxis) had 0 treated bleeds (n=18) (95% CI: 0.1; 27.3)1

HAVEN 2: CHILDREN WITH
FVIII INHIBITORS

HEMLIBRA® (emicizumab-kxwh) HAVEN 1-4 Long-term Data

of patients taking HEMLIBRA ONCE A WEEK had 0 treated bleeds (n=65) (95% CI: 64.8; 86.5)1,4

HEMLIBRA® (emicizumab-kxwh) HAVEN 1-4 Long-term Data

of patients taking HEMLIBRA ONCE EVERY 2 WEEKS had 0 treated bleeds (n=10)
(95% CI: 34.8; 93.3)3,5

HEMLIBRA® (emicizumab-kxwh) HAVEN 1-4 Long-term Data

of patients taking HEMLIBRA ONCE EVERY 4 WEEKS had 0 treated bleeds (n=10)
(95% CI 26.2; 87.8)3,5

HAVEN 3: ADULTS AND ADOLESCENTS WITHOUT FVIII INHIBITORS

HEMLIBRA® (emicizumab-kxwh) HAVEN 1-4 Long-term Data

of patients taking HEMLIBRA ONCE A WEEK had 0 treated bleeds (n=36) (95% CI: 38.1; 72.1)1

HEMLIBRA® (emicizumab-kxwh) HAVEN 1-4 Long-term Data

of patients taking HEMLIBRA ONCE EVERY 2 WEEKS hed 0 treated bleeds (n=35) (95% CI: 42.1; 76.1)1

HEMLIBRA® (emicizumab-kxwh) HAVEN 1-4 Long-term Data

of patients taking FVIll to treat bleeds on-demand (no prophylaxis) had 0 treated bleeds (n=18) (95% CI: 0; 18.5)1

HAVEN 4: ADULTS AND ADOLESCENTS WITH OR WITHOUT FVIII INHIBITORS

HEMLIBRA® (emicizumab-kxwh) HAVEN 1-4 Long-term Data

of patients taking HEMLIBRA ONCE EVERY 4 WEEKS had 0 treated bleeds (n=41) (95% CI: 39.7; 71.5)1

HEMLIBRA® (emicizumab-kxwh) HAVEN 1-4 Long-term Data

Median ABR for treated bleeds across all dosing schedules and populations receiving HEMLIBRA prophylaxis in HAVEN 1-4*1,3,4

HAVEN 1: 1.5 mg/kg QW. Mean ABRs*† (95% Cl): 2.9 (1.7; 5.0); no prophylaxis 23.3 (12.3; 43.9). Median time on treatment: 29 weeks; 24 weeks (no prophylaxis).
HAVEN 2: 1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W. Mean ABRs*† (95% Cl): 0.3 (0.2; 0.4); 0.2 (0.1; 0.5); 1.8 (0.3; 10.6). Median time on treatment: 58 weeks; 67 weeks; 67 weeks.
HAVEN 3: 1.5 mg/kg QW or 3 mg/kg Q2W. Mean ABRs*† (95% Cl): 1.5 (0.9; 2.5); 1.3 (0.8; 2.3); no prophylaxis 38.2 (22.9; 63.8). Median time on treatment: 30 weeks; 31 weeks; 24 weeks (no prophylaxis).
HAVEN 4: 6 mg/kg Q4W. Mean ABR*† (95% Cl): 2.4 (1.4; 4.3). Median time on treatment: 26 weeks.1,3-5
Maintenance doses shown here. Loading dose of HEMLIBRA is 3 mg/kg once weekly for the first 4 weeks, followed by the maintenance dose.1
Data from HAVEN 2 analysis are descriptive and therefore should be interpreted with caution.

*ABRs shown are for treated bleeds.
†ABRs were calculated with a negative binomial regression model, accounting for the difference in follow-up times.

Learn more about the side effects associated with HEMLIBRA.

HAVEN 1-4 LONG-TERM DATA

A POOLED ANALYSIS SUGGESTS: IMPROVEMENTS WITH HEMLIBRA OBSERVED IN THE PRIMARY ANALYSIS WERE SUSTAINED14

PERCENTAGE OF PATIENTS WITH ZERO TREATED BLEEDS MEASURED IN DISCRETE 24-WEEK INTERVALS OVER 144 WEEKS14

  • The ABR‡II (95% CI) over the entire study was 1.4 (1.1; 1.7). Median efficacy period (IQR) of 120.4 weeks (89.0; 164.4)14
  • No new safety concerns were identified. The most common treatment-related adverse reaction was ISR (107/399; 27%). The majority of all ISRs (treatment-related or not) were mild (104/111; 94%) and occurred during the first 24 weeks (93/111; 84%)14
  • There were 3 TMAs and 4 TEs. All 3 TMAs and 2 of 4 TEs were associated with concomitant aPCC use and were previously reported in HAVEN 114

‡ABRs shown are for treated bleeds.
§Based on the calculated mean ABR for bleeds treated with coagulation factors.
IIModel-based ABRs were calculated with a negative binomial regression model, accounting for the difference in follow-up times.
n=number of patients who contributed data for analyses at each time interval.

Why do groups differ in size?

Patients in these trials could choose to convert to commercial drug (available for patients with inhibitors in the US as of November 2017 and patients without inhibitors in the US as of October 2018) at any time. Once a patient started commercial therapy, they were excluded from subsequent treatment intervals. At the time of this analysis (May 2020), many patients had converted to commercially available HEMLIBRA.

These results include patients from all study arms, including those who crossed over to HEMLIBRA from the no-prophylaxis arm.

These groups included patients who discontinued treatment for other reasons.

These data are a descriptive analysis and therefore should be interpreted with caution.

Additional Clinical Trials of HEMLIBRA

HEMLIBRA® (emicizumab-kxwh) Clinical Trials Haven 6 Icon

Patients with mild or moderate hemophilia A without inhibitors
(n=72)
1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W8

HEMLIBRA® (emicizumab-kxwh) Clinical Trials Haven 7 Icon

Infants without inhibitors
(n=55)
1.5 mg/kg QW, 3 mg/kg Q2W, or 6 mg/kg Q4W9

See the Results

HEMLIBRA® (emicizumab-kxwh) Clinical Trials Haven Phase 1/2 Study Icon

Adults and adolescents with or without inhibitors
(n=18)
Dose-finding study6

HEMLIBRA® (emicizumab-kxwh) Clinical Trials Haven 5 Icon

Adults, adolescents, and children with or without inhibitors
(n=85)
1.5 mg/kg QW or 6 mg/kg Q4W11

HEMLIBRA® (emicizumab-kxwh) Clinical Trials HOHOEMI Icon

Children without inhibitors
(n=13)
3 mg/kg Q2W or 6 mg/kg Q4W7

See the Results

HEMLIBRA® (emicizumab-kxwh) Clinical Trials STASEY Icon

Adults and adolescents with inhibitors
(n=193)
1.5 mg/kg QW10

Expanding the experience

Outside of the HAVEN 1-4 pivotal trials, HEMLIBRA has also been widely studied in the real-world setting, leading to a total of more than 190 publications. See additional populations and clinical scenarios captured below:

  • Major and minor surgeries16,17
  • Infants <12 months without inhibitors9
  • Patients ≥65 years of age18
  • Females8,16
  • Tolerized patients or historical inhibitors20,21
  • Anti-drug antibodies21,22

A literature search of PubMed, ProQuest, and manual searches of major hemophilia congress abstract books was conducted on September 15, 2022. Search terms included “emicizumab,” “HEMLIBRA,” “ACE910,” and “RG6013.” Reports describing efficacy, safety, or surgical outcomes related to emicizumab in the real-world setting were selected. Some publications may contain duplicate patient participants who were assessed in more than one study.
FVIII=factor VIII; QW=once weekly; Q2W=once every 2 weeks; Q4W=once every 4 weeks.
ABR=annualized bleed rate; aPCC=activated prothrombin complex concentrate; CI=confidence interval; IQR=interquartile range; ISR=injection site reaction; TE=thrombotic event; TMA=thrombotic microangiopathy.
Maintenance doses shown here.
Other than Phase 1/2 study, all other studies evaluated approved HEMLIBRA doses.

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In Patients Without FVIII Inhibitors

Learn more about how HEMLIBRA works for adults and adolescents without FVIII inhibitors.

See the Results

In Patients With FVIII Inhibitors

Learn more about how adults and adolescents with FVIII inhibitors achieved zero treated bleeds.

See the Results
NEXT: Patients Without FVIII Inhibitors

Indication & Important Safety Information

Indication
HEMLIBRA is indicated for routine prophylaxis to prevent or reduce the frequency of bleeding episodes in adult and pediatric patients ages newborn and older with hemophilia A with or without factor VIII inhibitors.

Boxed WARNING: THROMBOTIC MICROANGIOPATHY and THROMBOEMBOLISM
Cases of thrombotic microangiopathy and thrombotic events were reported when on average a cumulative amount of >100 U/kg/24 hours of activated prothrombin complex concentrate (aPCC) was administered for 24 hours or more to patients receiving HEMLIBRA prophylaxis. Monitor for the development of thrombotic microangiopathy and thrombotic events if aPCC is administered. Discontinue aPCC and suspend dosing of HEMLIBRA if symptoms occur. 

Warnings and Precautions
Thrombotic Microangiopathy (TMA) and Thromboembolism Associated With HEMLIBRA and aPCC
In clinical trials, TMA was reported in 0.8% of patients (3/391) and thrombotic events were reported in 0.5% of patients (2/391). In patients who received at least one dose of aPCC, TMA was reported in 8.1% of patients (3/37) and thrombotic events were reported in 5.4% of patients (2/37). Patients with TMA presented with thrombocytopenia, microangiopathic hemolytic anemia, and acute kidney injury, without severe deficiencies in ADAMTS13.

Consider the benefits and risks if aPCC must be used in a patient receiving HEMLIBRA prophylaxis. Due to the long half-life of HEMLIBRA, the potential for an interaction with aPCC may persist for up to 6 months after the last dose. Monitor for the development of TMA and/or thromboembolism when administering aPCC. Immediately discontinue aPCC and interrupt HEMLIBRA prophylaxis if clinical symptoms, imaging, or laboratory findings consistent with TMA and/or thromboembolism occur, and manage as clinically indicated. Consider the benefits and risks of resuming HEMLIBRA prophylaxis following complete resolution of TMA and/or thrombotic events on a case-by-case basis.

Immunogenicity
Treatment with HEMLIBRA may induce anti-drug antibodies. Anti-emicizumab-kxwh antibodies were reported in 5.1% of patients (34/668) treated with HEMLIBRA in clinical trials. Most patients with anti-emicizumab-kxwh antibodies did not experience a change in HEMLIBRA plasma concentrations or an increase in bleeding events; however, in uncommon cases (incidence <1%), the presence of neutralizing antibodies with decreasing plasma concentration may be associated with loss of efficacy.

Monitor for clinical signs of loss of efficacy (eg, increase in breakthrough bleeding events) and if observed, promptly assess the etiology and consider a change in treatment if neutralizing anti-emicizumab-kxwh antibodies are suspected.

Laboratory Coagulation Test Interference
HEMLIBRA affects intrinsic pathway clotting-based laboratory tests, including activated clotting time (ACT); activated partial thromboplastin time (aPTT); and all assays based on aPTT, such as one-stage, factor VIII (FVIII) activity. Therefore, intrinsic pathway clotting-based coagulation laboratory test results in patients who have been treated with HEMLIBRA prophylaxis should not be used to monitor HEMLIBRA activity, determine dosing for factor replacement or anti-coagulation, or measure FVIII inhibitor titers.

Results affected by HEMLIBRA: aPTT; Bethesda assays (clotting-based) for FVIII inhibitor titers; one-stage, aPTT-based single-factor assays; aPTT-based Activated Protein C Resistance (APC-R); ACT.

Results unaffected by HEMLIBRA: Bethesda assays (bovine chromogenic) for FVIII inhibitor titers; thrombin time (TT); one-stage, prothrombin time (PT)-based single-factor assays; chromogenic-based single-factor assays other than FVIII (see Drug Interactions for FVIII chromogenic activity assay considerations); immuno-based assays (ie, ELISA, turbidimetric methods); genetic tests of coagulation factors (eg, Factor V Leiden, Prothrombin 20210).

Most Common Adverse Reactions
The most common adverse reactions (incidence ≥10%) are injection site reactions, headache, and arthralgia.

Adverse Reactions
Characterization of aPCC Treatment in Pooled Clinical Trials
There were 130 instances of aPCC treatment in 37 patients, of which 13 instances (10%) consisted of on average a cumulative amount of >100 U/kg/24 hours of aPCC for 24 hours or more; 2 of the 13 were associated with thrombotic events and 3 of the 13 were associated with TMA. No TMA or thrombotic events were associated with the remaining instances of aPCC treatment.

Injection Site Reactions
In total, 85 patients (22%) reported injection site reactions (ISRs). All ISRs observed in HEMLIBRA clinical trials were reported as mild to moderate intensity and 93% resolved without treatment. The commonly reported ISR symptoms were injection site erythema (11%), injection site pruritus (4%), and injection site pain (4%).

Other Less Common (<1%) Reactions
Rhabdomyolysis was reported in 2 adult patients with asymptomatic elevations in serum creatine kinase without associated renal or musculoskeletal symptoms. In both instances, the event occurred following an increase in physical activity.

Drug Interactions
Clinical experience suggests that a drug interaction exists with HEMLIBRA and aPCC.

Pregnancy, Lactation, Females and Males of Reproductive Potential
Women of childbearing potential should use contraception while receiving HEMLIBRA. It is not known whether HEMLIBRA can cause fetal harm when administered to a pregnant woman or can affect reproduction capacity. HEMLIBRA should be used during pregnancy only if the potential benefit for the mother outweighs the risk to the fetus. The developmental and health benefits of breastfeeding should be considered along with the mother’s clinical need for HEMLIBRA and any potential adverse effects on the breastfed child from HEMLIBRA or from the underlying maternal condition.

You may report side effects to the FDA at (800) FDA-1088 or www.fda.gov/medwatch. You may also report side effects to Genentech at (888) 835-2555.

Please see the HEMLIBRA full Prescribing Information for additional Important Safety Information, including Boxed WARNING.

    • HEMLIBRA package insert. South San Francisco, CA: Genentech, Inc.; 2024.

      HEMLIBRA package insert. South San Francisco, CA: Genentech, Inc.; 2024.

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    • Data on File. Genentech, Inc.

      Data on File. Genentech, Inc.

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